COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING
摘要
<p>Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.</p>
申请公布号
WO2010120820(A1)
申请公布日期
2010.10.21
申请号
WO2010US30940
申请日期
2010.04.13
申请人
ISIS PHARMACEUTICALS, INC.;COLD SPRING HARBOR LABORATORY;KRAINER, ADRIAN, R.;HUA, YIMIN;BAKER, BRENDA, F.;FREIER, SUSAN, M.;BENNETT, C., FRANK
发明人
KRAINER, ADRIAN, R.;HUA, YIMIN;BAKER, BRENDA, F.;FREIER, SUSAN, M.;BENNETT, C., FRANK
